Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Microvascular perfusion
研究概览
简要总结
The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of the heart and kidney. To test this question, the investigators will perform imaging tests (contrast ultrasound perfusion imaging) to look at the flow and function of these microvessels and compare this information to heart and kidney function. To further look at this question, patients who have PNH will be studied before and after starting a new drug (Soliris) that decreases damage to blood cells. In patients with sickle cell disease, patients will be studied at baseline (not during a pain crisis) and also during a pain crisis if one develops.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 年龄范围
- 19 Years 至 —(Adult, Older Adult)
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Established diagnosis of PNH or SCD Age > 18 years old
排除标准
- •Pregnant or lactating women Presence of significant right to left shunting Allergy to ultrasound contrast agent Reactive airways disease Significant peripheral or coronary artery disease
结局指标
主要结局
Microvascular perfusion
时间窗: 1 hour
1. Myocardial microvascular blood flow (MBF) and capillary blood velocity (CBV) on study day. 2. Renal MBF on study day 3. Skeletal muscle MBF and CBV on study day 4. Myocardial MBF and CBV during hyperemia 5. Skeletal muscle CBV during hyperemia
次要结局
未报告次要终点
研究者
Jonathan R. Lindner, MD
Professor
Oregon Health and Science University
